Novel RNA-based strategies for therapeutic gene silencing

Christopher R Sibley, Yiqi Seow, Matthew J A Wood

Research output: Contribution to journalReview articlepeer-review

Abstract / Description of output

The past decade has seen intense scientific interest in non-coding RNAs. In particular, the discovery and subsequent exploitation of gene silencing via RNA interference (RNAi) has revolutionized the way in which gene expression is now studied and understood. It is now well established that post-transcriptional gene silencing (PTGS) by the microRNA (miRNA) and other RNAi-associated pathways represents an essential layer of complexity to gene regulation. Gene silencing using RNAi additionally demonstrates huge potential as a therapeutic strategy for eliminating pathogenic gene expression. Yet despite the early promise and excitement of gene-specific silencing, several critical hurdles remain to be overcome before widespread clinical adoption. These include off-target effects, toxicity due to saturation of the endogenous RNAi functions, limited duration of silencing, and effective targeted delivery. In recent years, a range of novel strategies for producing RNA-mediated silencing have been developed that can circumvent many of these hurdles, including small internally segmented interfering RNAs, tandem hairpin RNAs, and pri-miRNA cluster mimics. This review discusses RNA-mediated silencing in light of this recent research, and highlights the benefits and limitations conferred by these novel gene-silencing strategies.

Original languageEnglish
Pages (from-to)466-476
Number of pages11
JournalMolecular Therapy
Issue number3
Early online date14 Dec 2016
Publication statusE-pub ahead of print - 14 Dec 2016

Keywords / Materials (for Non-textual outputs)

  • animals
  • gene expression
  • gene silencing
  • genetic techniques
  • genetic therapy
  • genetics
  • humans
  • RNA Interference
  • models
  • RNA/genetics


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